This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Subcutaneous Immunotherapy for Mouse in Adults (NCT02532179), a na phase 1, phase 2 study. The primary outcome is Number of Reported Adverse Events (AEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02532179 |
| Phase | Phase 1, Phase 2 |
| Condition | Asthma, Perennial Allergic Rhinitis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 12 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Institute of Allergy and Infectious Diseases (NIAID) |
| Results posted | Yes |
2. Clinical Question
The clinical question concerns an early-stage study of mouse allergenic extract given by subcutaneous injection to adults with mouse sensitivity and asthma and/or perennial allergic rhinitis. The registry record describes a pilot designed to assess safety, immune-response biomarkers, and whether the therapy might be effective in treating allergic asthma. The posted primary outcome is a count of reported adverse events, rather than a comparative measure of clinical benefit.
Population
Adults described as mouse-sensitive, with asthma and/or perennial allergic rhinitis. The primary outcome population is listed as intent-to-treat. The registry record lists an enrollment of 12.
Intervention
Mouse allergenic extract administered by subcutaneous injection. The arm description specifies escalating doses up to a maximum study dose of 0.4 mL at a concentration of 1:10 wt/vol.
Comparator
No separate comparator group is listed. The design is single-group and open label, so the record does not present a like-for-like control comparison.
Primary question
The primary outcome is the number of reported adverse events, tabulated by preferred event term, from baseline before treatment through 24 weeks of treatment. The outcome result itself is not reported in the registry record.
3. Trial Design
The registry record identifies this as a Phase 1 and Phase 2, open-label, single-group pilot study. Allocation is listed as not applicable, and no masking is listed. Enrollment was 12. The record lists the study as completed, with a start in 2015-10 and primary completion in 2016-10.
Participants received mouse allergenic extract by subcutaneous injection. The arm description says that doses escalated up to a maximum study dose of 0.4 mL of extract at a concentration of 1:10 wt/vol. The record does not specify the dose-escalation schedule, the timing of individual injections, or criteria used to change or stop dosing; those details are not reported in the registry record.
The brief summary describes three aims: evaluating safety, examining immune-response biomarkers, and assessing whether the therapy might be effective in allergic asthma. The listed secondary outcomes concern changes in mouse-specific IgE, IgG, and IgG4 antibodies, and in-vitro mouse-antigen binding to B-cells. The registry lists six outcome measures and three analyses, but the details of those analyses are not reported in the registry record.
Because the study has one group and no listed control, its design can describe observations during treatment but does not provide a randomized or concurrent comparison. The record does not report a sample-size justification, detailed eligibility criteria, or a statistical analysis plan. Any planning calculations based on an observed effect should therefore be read as illustrations, not as evidence that the study was designed or powered to detect that effect.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Mouse Allergenic Extract |
|---|---|
| STARTED | 12 |
| COMPLETED | 9 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | Mouse Allergenic Extract |
|---|---|
| Lost to Follow-up | 1 |
| Pregnancy | 1 |
| Subject received depot steroid injection | 1 |
Analysis population for the primary outcome, as defined in the registry: Intent-to-treat
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Mouse Allergenic Extract |
|---|---|---|
| Participants analysed | 12 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 12 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 27.3 (SD 7.54) |
| Sex: Female, Male: Female | Participants | 8 |
| Sex: Female, Male: Male | Participants | 4 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 10 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 9 |
| Race (NIH/OMB): White | Participants | 2 |
| Race (NIH/OMB): More than one race | Participants | 1 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 12 |
| Mouse-Specific Serum Immunoglobulin E (IgE) Levels | kU/L | 2.34 (SD 0.45) |
| Mouse-Specific Serum Immunoglobulin G (IgG) Levels | kU/L | 1.23 (SD 0.20) |
| Mouse-Specific Serum Immunoglobulin G4 (IgG4) Levels | kU/L | 0.08 (SD 0.35) |
6. Primary Endpoint
The registry lists the primary outcome as Number of Reported Adverse Events (AEs), measured over Baseline (Pre-Treatment) through 24 Weeks of Treatment.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Events |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is titled Number of Reported Adverse Events (AEs). It is measured as a number of events, with frequency tabulated by preferred event term. The stated observation period runs from baseline before treatment through 24 weeks of treatment, and the listed population is intent-to-treat.
A count outcome records how many events are reported, rather than measuring the proportion of participants who experience at least one event. Those quantities answer different questions: one participant can potentially contribute more than one event, while a participant-level affected count records whether participants experienced an event. The posted adverse-event summary includes participant counts, but the primary endpoint result—the event count by preferred term—is not reported in the registry record. The participant counts should not be substituted for that missing primary outcome result.
Adverse-event counts are used in safety reporting because they describe the events observed during a specified period. Their interpretation depends on what was counted, how events were classified, and how repeated events were handled. The registry record gives the preferred-term approach and timeframe, but does not report event-level counts or further counting rules. The endpoint alone does not establish that an event was caused by treatment; the record does not report a causal assessment method.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The available record supports a limited descriptive reading. It identifies a completed, open-label, single-group study with an enrollment of 12 and a primary outcome focused on reported adverse events during the period from baseline before treatment through 24 weeks of treatment. It does not report the primary event-count result, so the frequency by preferred event term cannot be assessed from the registry record.
The posted safety summary lists 12 participants at risk for serious adverse events and a serious-event affected count of 0. It lists 12 participants at risk for other adverse events and an affected count of 6. No deaths are listed, and no top serious-event terms are listed. These are descriptive registry figures. The serious-event affected count is not the same measure as a complete event count, and the other-event participant count does not indicate how many events occurred or what those events were. The record does not report the event terms for the other adverse events.
These counts should be interpreted within the design. With one open-label group and no listed comparator, the results cannot separate events occurring during treatment from events that might also have occurred without treatment. Nor can they support a like-for-like estimate of a treatment contrast. The registry record does not report an independent event-adjudication process or a causal attribution method.
No effect estimate, confidence interval, or p-value for the primary outcome is reported in the registry record. A confidence interval, in general, conveys the precision of an estimate; without an endpoint estimate and its interval, the precision of the primary count cannot be evaluated. The registry record also does not report the results of the listed biomarker outcomes or a clinical efficacy outcome. The study aims mention biomarkers and possible efficacy, but an aim is not a reported result.
The registry describes six outcome measures and three analyses, but does not report enough detail to establish which analysis corresponds to each measure, whether any analysis was adjusted, or how missing observations were handled. Any interpretation should therefore stay close to the posted descriptive information. The figures do not establish safety in a broader population, comparative effectiveness, or a treatment effect.
9. Statistical Methods Explained
Understanding a single-group design
In a single-group study, participants receive the intervention and outcomes are observed in that group. This can provide descriptive information about what was recorded during the study. Without a separate comparison group, however, there is no concurrent estimate of what would have happened under another treatment or without the study intervention. General statistical principle: a before-and-after observation is not by itself a controlled treatment contrast, because changes can reflect factors other than the intervention.
Here, the registry lists allocation as not applicable, masking as none, and the model as single group. It describes an open-label study of subcutaneous mouse allergenic extract. The registry record does not report a randomized comparison or a control arm. Accordingly, the adverse-event figures describe the enrolled group and should not be interpreted as a comparative risk estimate.
Interpreting an event count
The primary outcome is a count of reported adverse events, tabulated by preferred event term. Counts and participant counts are not interchangeable. A count of events can include multiple events associated with one participant, whereas an affected-participant count indicates how many participants experienced at least one event of the relevant category. The posted safety summary gives affected counts, but the primary outcome’s event counts by term are not reported in the registry record.
General statistical principle: count data are often unevenly distributed, with many observations at low counts and fewer at high counts. Some count models assume that variability follows a particular relationship to the average count. If the actual variability is greater than the model assumes, a condition called over-dispersion, standard errors can be too small and intervals or tests can appear more precise than warranted. This is a general consideration, not a statement about an analysis used in this trial. The registry record does not report the model used for the primary endpoint or whether over-dispersion was assessed.
Event counting also depends on operational choices: what qualifies as an event, whether repeated occurrences are counted separately, how terms are coded, and how follow-up time is handled. The record says events are tabulated by preferred event term and gives a timeframe from baseline before treatment through 24 weeks of treatment. Other event-counting rules are not reported in the registry record.
Estimates, uncertainty, and tests
A point estimate is a single numerical summary of an outcome. A confidence interval, in general, gives a range of values compatible with the data and the method used, and communicates uncertainty around an estimate. A p-value, in general, describes how unusual the data would be under a stated statistical null model; it is not a measure of clinical importance. The registry record does not report a primary endpoint estimate, confidence interval, or p-value. Therefore, neither the magnitude nor the statistical precision of the primary event-count outcome can be evaluated from the reported information.
The sample-size assumptions describe planning illustrations based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Those are hypothetical planning assumptions, not the design of this single-group study. The illustration is not observed power, and its assumed two-arm allocation does not turn the actual study into a controlled comparison. The registry record does not report the trial’s own sample-size justification.
Outcome families and multiplicity
The registry lists six outcome measures, including the primary adverse-event measure and secondary antibody and B-cell binding outcomes, and lists three analyses. When several outcomes or analyses are considered, the chance of at least one apparently notable result can be affected by how many statistical questions are tested and by the dependence among them. General statistical principle: multiplicity methods can control or otherwise address that issue, but the appropriate method depends on the analysis plan and goals. The registry record does not report whether multiplicity adjustments were planned or used, nor how the three analyses relate to the outcome measures.
Analysis populations and missing observations
The primary outcome population is identified as intent-to-treat. That label generally refers to analyzing participants according to the group to which they were assigned, but this study has no listed randomized allocation or comparison group. The registry record does not give a more detailed definition of the intent-to-treat population or explain how participants with incomplete follow-up were handled.
Missing data can affect estimates when participants do not contribute all planned observations. General statistical principle: the impact depends on why values are missing, how much information is missing, and the analysis method. The registry record does not report the amount or pattern of missing outcome data, any imputation method, or sensitivity analyses. These omissions limit assessment of the completeness and robustness of the reported results.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists six outcome measures and three analyses. The named secondary outcomes concern changes in mouse-specific IgE, IgG, and IgG4 antibodies, and in-vitro mouse-antigen binding to B-cells. The primary outcome concerns adverse-event counts. A set of related outcomes can create multiplicity: when several questions are tested, the overall chance of a false-positive finding may differ from the chance for one prespecified test. General statistical approaches can address multiplicity, but the choice depends on the analysis plan. Whether any adjustment, hierarchy, or prespecified testing order was used is not reported in the registry record.
The primary outcome population is listed as intent-to-treat. The registry record does not provide an operational definition for this population, explain how incomplete follow-up was handled, or report the number of participants contributing data to each outcome. The record also does not report missing-data methods, imputation, or sensitivity analyses. Consequently, the completeness of the outcome data and the effect of missing observations cannot be assessed.
Although the study is listed as single-group, the sample-size planning assumptions refer to equal allocation between two arms and a normal approximation. They are planning illustrations based on an observed effect, not the trial’s own design or observed power. The registry record does not report the protocol’s sample-size justification, interim analyses, or a statistical analysis plan. It is therefore not possible to determine from the registry record whether the analyses followed a prespecified plan or how any multiplicity and missing-data issues were addressed.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Mouse Allergenic Extract | 0 | 12 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Mouse Allergenic Extract | 0 of 12 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Mouse Allergenic Extract | 6 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Mouse Allergenic Extract |
|---|---|---|
| Injection site swelling | General disorders | 2 of 12 |
| Peak expiratory flow rate decreased | Investigations | 2 of 12 |
| Abdominal Pain | Gastrointestinal disorders | 1 of 12 |
| Vomiting | Gastrointestinal disorders | 1 of 12 |
| Pruritus generalised | Skin and subcutaneous tissue disorders | 1 of 12 |
| Skin lesion | Skin and subcutaneous tissue disorders | 1 of 12 |
| Flushing | Vascular disorders | 1 of 12 |
The posted safety summary lists 12 participants at risk for serious adverse events and a serious-event affected count of 0. It lists 12 participants at risk for other adverse events and an affected count of 6. The listed death count is 0, and no top serious-event terms are listed.
These figures are participant-level affected counts in the safety summary, not a complete count of all individual adverse events. They do not show how many events occurred per affected participant, the terms of the other adverse events, or whether the events were judged related to treatment. The primary endpoint is a count of events tabulated by preferred term, but that result is not reported in the registry record.
The absence of a listed serious-event affected participant in this group is a descriptive finding for this study, not proof that serious events cannot occur. The study enrolled 12 participants, had no listed comparator, and was open label. The registry record does not provide a comparative safety estimate, detailed event narratives, or a causal attribution method. These limits should remain part of any reading of the safety figures.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a completed pilot study evaluated subcutaneous mouse allergenic extract in adults with mouse sensitivity and asthma and/or perennial allergic rhinitis. It identifies safety and immune-response biomarkers among the study aims and gives a primary outcome focused on the number of reported adverse events over the stated treatment period.
The posted safety summary reports a serious-event affected count of 0 among 12 at risk, an other-adverse-event affected count of 6 among 12 at risk, and a death count of 0. These are descriptive participant counts. They do not provide the primary endpoint’s event counts by preferred term, and they do not specify the types of other adverse events. The primary endpoint result is not reported in the registry record.
The record does not report results for the secondary biomarker outcomes or a clinical efficacy outcome. Thus, the stated aims to examine immune response and possible efficacy cannot be treated as evidence that those outcomes changed or that a clinical benefit was demonstrated. The registry record also does not report an effect estimate, confidence interval, or p-value for the primary outcome.
The study’s single-group, open-label design is important to interpretation. It can describe observations made among participants receiving the extract, but it does not offer a like-for-like control comparison. The information does not establish that reported events were caused by treatment, nor can it estimate a comparative treatment effect. General statistical principle: findings from a small pilot can inform what to examine in later research, but they do not by themselves establish how well results generalize to other populations or settings.
Accordingly, the most defensible reading is descriptive and limited to the recorded study group. The registry record does not establish comparative effectiveness or rule out safety risks beyond the observations recorded.
13. Why This Trial Matters Statistically
This study is statistically instructive because it distinguishes an early-stage, single-group safety and biomarker investigation from a controlled efficacy comparison. The registry describes aims spanning safety, immune response, and possible efficacy, but the primary outcome is an adverse-event count. Keeping the stated aim separate from the reported endpoint helps prevent a proposed question from being mistaken for a result.
It also illustrates why outcome units matter. The primary outcome is a count of events by preferred term, while the safety summary reports affected-participant counts. Those summaries are not interchangeable: a participant-level count cannot show the total number of events or their distribution across event terms. The primary count result itself is not reported in the registry record.
The single-group, open-label structure makes the distinction between description and comparison especially important. Event observations can be described within the study group, but no concurrent control group is listed to provide a comparative benchmark. A finding observed during treatment therefore should not automatically be attributed to treatment.
The registry also lists multiple outcome measures and analyses without describing the analysis plan, missing-data handling, or any multiplicity approach. That makes it difficult to assess how the various statistical questions were prioritized or how uncertainty was handled. Planning illustrations based on an observed effect do not establish observed power or substitute for a protocol-based sample-size justification.
Together, these features make the record useful for understanding what was studied and what kinds of outcomes were considered, while underscoring how design, outcome definition, and reporting detail constrain statistical conclusions.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
Several limitations arise directly from the trial description and the information registered. The study is open label and single group, with no separate comparator listed. This limits interpretation to descriptions of the enrolled group and does not permit a like-for-like controlled comparison. The registry record does not describe blinding or a method for independent event assessment.
Enrollment was 12. The record does not report a protocol-based sample-size justification, so the intended precision or design rationale cannot be evaluated. Planning illustrations based on an observed effect use assumptions that include equal allocation between two arms, which do not describe the listed single-group design. Such illustrations are not observed power.
The primary outcome is specified as an event count by preferred term, but its result is not reported in the registry record. The safety summary gives participant counts, not the primary event-count detail. It also does not provide the types of other adverse events or a causal assessment. Results for the listed biomarker outcomes are not reported in the registry record.
The registry record does not report a detailed statistical analysis plan, the relationship between the six outcome measures and three analyses, multiplicity handling, missing-data amounts or methods, or sensitivity analyses. It also does not report the detailed dosing schedule or other event-counting rules. These gaps limit evaluation of analysis choices, completeness, and precision. The results should therefore be read as limited registry descriptions rather than as a full statistical account of the study.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Subcutaneous Immunotherapy for Mouse in Adults — NCT02532179.
- Registry record last updated 2018-03-07; status last verified 2018-02.
- Results first posted 2017-11-28.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT02532179 is a completed Phase 1 and Phase 2, open-label, single-group pilot of subcutaneous mouse allergenic extract in adults with mouse sensitivity and asthma and/or perennial allergic rhinitis. Enrollment was 12. The arm description specifies escalating doses up to 0.4 mL at a concentration of 1:10 wt/vol.
The primary outcome is the number of reported adverse events by preferred term from baseline before treatment through 24 weeks of treatment, in the intent-to-treat population. Its result is not reported in the registry record. The safety summary lists 0 participants affected by serious adverse events, 6 affected by other adverse events, and 0 deaths. The record lists secondary biomarker outcomes, but their results are not reported in the registry record.
The study has no listed comparator. The registry record does not report the detailed statistical analysis plan, multiplicity or missing-data methods, or a sample-size justification. The findings are therefore limited to the descriptive information recorded for this small, single-group study.